CRISPR Technologies for In Vivo and Ex Vivo Gene Editing

Abstract

What Is the Issue? The first therapeutics based on clustered regularly interspaced short palindromic repeats (CRISPR) technologies are entering the market. These gene editing technologies have the potential to change treatment paradigms and may be used to treat conditions that cannot be treated or cured with current methods. This report aims to provide an overview of the technologies and their current and potential roles in health care. What Are the Technologies? CRISPR is a part of bacterial immune systems that can cut DNA strands and is used as a gene editing tool. A guide ribonucleic acid (RNA) sequence leads the CRISPR-associated nuclease to the target DNA sequence where the cut is made. These edits change the function of the gene, making genes nonfunctional or replacing the coding sequence for 1 gene with another. CRISPR can also be used to increase or decrease the expression of specific genes. What Is the Potential Impact? CRISPR-based technologies have a variety of potential applications in health care, including: treating genetic diseases understanding the genetic mechanisms of diseases and investigating the relevance of potential drug treatments managing infectious diseases through detection, treatment, and elimination. What Else Do We Need to Know? Ethical issues pertinent to the use of CRISPR include the ability to obtain adequately informed consent, the potential future consequences of gene editing and its potential unintended effects, and the impact gene editing could have on future generations. The long-term effects of CRISPR-based therapies are currently unknown. Further research into emerging applications is required. Long-term follow-up of the patients who have received the first CRISPR-based therapeutics will help inform understanding of the safety and effectiveness of these treatments. While the first of these therapies have been granted regulatory authorization, the next viable CRISPR-based therapies are still in the early phases of development, with the pivotal clinical trials not expected to be completed until at least 2027.